Kamada has announced that still plans to hold discussions with the EMA and FDA regarding regulatory submissions for its inhaled Alpha-1 antitrypsin (AAT) even after the drug failed to meet either its primary or secondary endpoints in a Phase 2/3 clinical study of inhaled AAT for the treatment of Alpha-1 antitrypsin deficiency (AATD).
The company said that inhaled AAT showed no statistically significant difference from placebo in time to first moderate or severe exacerbation, the primary endpoint, as announced earlier this year, and that final analysis showed that the drug also failed to meet secondary endpoints, “time to first event-based exacerbation with a severity of mild, moderate or severe” and “severity of the first exacerbation event.”
However, Kamada said, FEV1 and other lung function parameters “which were collected to support safety endpoints, showed concordance of a potential treatment effect in the reduction of the inflammatory injury to the lung that is known to be associated with a reduced loss of respiratory function.” The company also noted that intravenous AAT therapies were approved based on PK and safety data alone.
Kamada Co-Founder and CEO David Tsur commented, “We strongly believe the clinically meaningful differences seen in lung function parameters are therapeutically relevant, particularly with regard to the most frequent exacerbators, who have the greatest need for effective new therapies and comprise a major portion of this orphan patient population. Based on orphan designation of the drug, prior discussions held with the regulator, the strength of these data and the persistent unmet need in this orphan indication, we will advance our discussions with the European Medicines Agency with the intent to submit for conditional approval in order to bring our inhaled AAT to patients with AATD in Europe, and will initiate discussion with the FDA to determine a US path for registration.”
Tsur added, “We remain committed to AATD patients worldwide and to maintaining our leadership role in the development of innovative new therapies for this orphan lung disease. As such, we remain steadfast in conducting continued clinical work in support of our inhaled AAT, including our ongoing open-label extension study in Europe and Phase 2 US study, as well as other future studies that would support its global licensure and expand its use into other lung diseases. Moreover, these very promising data put us in an attractive position as we advance our ongoing discussions with partners for potential licensing opportunities in markets outside of Europe.”
Chiesi signed a 12-year agreement with Kamada in August 2012 for exclusive distribution of inhaled AAT in Europe, Turkey and former CIS countries. That agreement includes up to $60 million in milestone payments.
Read the Kamada press release.
